OBJECTIVES: The aim of this study is to conduct a cost minimization analysis (CMA) of prophylactic treatment of Emicizumab in inhibitor-negative patients with hemophilia A compared to prophylactic and episodic treatments with pdFVIIIs or rFVIIIs from the perspective of the national reimbursement agency (SSI) in Turkey.

METHODS: The study was conducted based on HAVEN3 clinical trial, which was designed to model the treatment of inhibitor-negative Hemophilia A patients. A cost minimization model was developed in Microsoft Excel®. In the base case analysis, Emicizumab prophylaxis was compared to prophylactic and episodic FVIII treatments. Probability of developing inhibitors to FVIII during Emicizumab treatment was calculated as 0,45% per year and included in the costs. The costs associated with the complications that may occur in patients with hemophilia A and the adverse events reported in the HAVEN3 trial were added to the direct costs. All direct medical costs were included in the model from SSI perspective considering a calendar year. Calculations were made in TRY and reported after converting into Euro (1€=6,67 TRY; 2 May 2019).

RESULTS: While the average direct cost per patient per year was calculated as €106.981,20 with Emicizumab prophylaxis for the treatment of inhibitor-negative patients with hemophilia A, it was calculated as €117.140,70 with FVIII prophylaxis and €404.385 with episodic treatment. Compared to episodic treatment (€ -297.404) and prophylactic treatment (€ -10.160) with FVIII, Emicizumab prophylaxis is found to be a less costly treatment option and to be a cost-minimizing treatment.

CONCLUSIONS: Prophylactic treatment of Emicizumab for inhibitor-negative patients with hemophilia A is a less costly treatment and is an option to reduce the cost burden to SSI compared to other treatments.